Editas Medicine, MGH Announce Agreement
News Aug 04, 2016
Editas Medicine, Inc. has announced that it has entered into an exclusive license agreement with Massachusetts General Hospital (MGH) to access intellectual property and technology related to high-fidelity Cas9 nucleases and Cas9 PAM variants that will enable the Company to address an expanded range of genetically-defined diseases with the potential for enhanced specificity.
“This agreement with MGH marks additional progress on our strategy of building a company committed to advancing the science behind CRISPR to benefit patients facing genetically-defined diseases,” said Katrine Bosley, President and Chief Executive Officer of Editas Medicine. “Keith Joung and his MGH team have done tremendous work in creating these novel, engineered forms of Cas9, and these advancements align fully with our highly differentiated genome editing platform. We are eager to deploy them and unlock their therapeutic potential.”
“I’m delighted to hear that MGH and Editas Medicine have entered into this important agreement,” said J. Keith Joung, M.D., Ph.D., Associate Chief for Research, the Jim and Ann Orr MGH Research Scholar in the MGH Department of Pathology, and a scientific co-founder of and consultant to Editas Medicine. “I look forward to the full potential of these high-fidelity Cas9 and Cas9 PAM variants being maximized to develop safe and effective therapies for patients with a broad range of diseases.”
As published in the January 2016 issue of Nature, MGH researchers led by Dr. Joung described their high-fidelity Cas9 variant – Streptococcus pyogenes Cas9-HF1 – designed to reduce non-specific DNA contacts. SpCas9-HF1 rendered all or nearly all off-target events undetectable by genome-wide break capture and targeted sequencing methods.
The MGH team has also identified and characterized a series of novel S. pyogenes and S. aureus Cas9 PAM variants that substantially increase the range of sites in the genome that can be targeted for genome editing. These PAM variants were described in more detail in the July 23, 2015 issue of Nature and the December 2015 issue of Nature Biotechnology. The protospacer adjacent motif, or PAM, is the region of the Cas9 protein that helps determine where Cas9 can bind to DNA.
In a new study in cells, University of Illinois researchers have adapted CRISPR gene-editing technology to cause the cell’s internal machinery to skip over a small portion of a gene when transcribing it into a template for protein building. This gives researchers a way not only to eliminate a mutated gene sequence, but to influence how the gene is expressed and regulated.